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Updated: Sep 20, 2026

Molecular and Immunologic Techniques in a Genetically Engineered Mouse Model of Gastrointestinal Stromal Tumor
07:21

Molecular and Immunologic Techniques in a Genetically Engineered Mouse Model of Gastrointestinal Stromal Tumor

Published on: May 2, 2022

[Target-based therapy against gastrointestinal stromal tumors--from molecular diagnosis to molecular target therapy]

Toshirou Nishida1, Keigo Yasumasa

  • 1Department of Surgery, E1, Osaka University Graduate School of Medicine, 2-2 Yamada-oka, Suita, Osaka 565-0871, Japan.

Gan to Kagaku Ryoho. Cancer & Chemotherapy
|August 27, 2003
PubMed

Insights

Gastrointestinal stromal tumors (GIST) are driven by KIT or PDGF-R alpha gene mutations. STI571 (Gleevec) effectively treats GIST, offering good safety, but drug resistance is an emerging challenge.

Area of Science:

  • Oncology
  • Molecular Biology
  • Gastroenterology

Context:

  • Gastrointestinal stromal tumors (GIST) originate from KIT-positive mesenchymal cells.
  • Causative genetic mutations include gain-of-function in c-kit (90%) or PDGF-R alpha (5%).

Purpose:

  • To evaluate the efficacy and safety of STI571 (Gleevec) for GIST treatment.
  • To highlight Gleevec as a pioneering molecular targeted therapy for solid tumors.

Summary:

  • STI571 (Gleevec) demonstrates significant efficacy (60% partial response) in non-resectable or relapsed GIST.
  • The drug exhibits favorable tolerability and safety with infrequent severe adverse effects.
  • Gleevec represents a breakthrough in molecular targeted therapy for solid tumors.

Impact:

  • Establishes STI571 (Gleevec) as a key therapeutic option for GIST.
  • Addresses the critical need for effective treatments in advanced GIST.
  • Underscores the emergence of drug resistance as a significant future challenge in GIST therapy.

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